Abstract
Gene therapy is a promising strategy for the treatment of several inherited and acquired human diseases. Several vector platforms exist for the delivery of therapeutic nucleic acids into cells. Vectors based on viruses are very efficient at introducing gene constructs into cells, but their use has been associated with genotoxic effects of vector integration or immunological complications due to repeated administration in vivo. Non-viral vectors are easier to engineer and manufacture, but their efficient delivery into cells is a major challenge, and the lack of their chromosomal integration precludes long-term therapeutic effects. Transposable elements are non-viral gene delivery vehicles found ubiquitously in nature. Transposon-based vectors have the capacity of stable genomic integration and long-lasting expression of transgene constructs in cells. Molecular reconstruction of Sleeping Beauty, an ancient transposon in fish, represents a cornerstone in applying transposition-mediated gene delivery in vertebrate species, including humans. This review summarizes the stateof- the-art in the application of transposable elements for therapeutic gene transfer, and identifies key targets for the development of transposon-based gene vectors with enhanced efficacy and safety for human applications.
Keywords: expression cassette, virus vectors, Tc1 transposon family, DNA Transposition, RNA interference
Current Gene Therapy
Title: Transposons for Gene Therapy!
Volume: 6 Issue: 5
Author(s): Zoltan Ivics and Zsuzsanna Izsvak
Affiliation:
Keywords: expression cassette, virus vectors, Tc1 transposon family, DNA Transposition, RNA interference
Abstract: Gene therapy is a promising strategy for the treatment of several inherited and acquired human diseases. Several vector platforms exist for the delivery of therapeutic nucleic acids into cells. Vectors based on viruses are very efficient at introducing gene constructs into cells, but their use has been associated with genotoxic effects of vector integration or immunological complications due to repeated administration in vivo. Non-viral vectors are easier to engineer and manufacture, but their efficient delivery into cells is a major challenge, and the lack of their chromosomal integration precludes long-term therapeutic effects. Transposable elements are non-viral gene delivery vehicles found ubiquitously in nature. Transposon-based vectors have the capacity of stable genomic integration and long-lasting expression of transgene constructs in cells. Molecular reconstruction of Sleeping Beauty, an ancient transposon in fish, represents a cornerstone in applying transposition-mediated gene delivery in vertebrate species, including humans. This review summarizes the stateof- the-art in the application of transposable elements for therapeutic gene transfer, and identifies key targets for the development of transposon-based gene vectors with enhanced efficacy and safety for human applications.
Export Options
About this article
Cite this article as:
Ivics Zoltan and Izsvak Zsuzsanna, Transposons for Gene Therapy!, Current Gene Therapy 2006; 6 (5) . https://dx.doi.org/10.2174/156652306778520647
DOI https://dx.doi.org/10.2174/156652306778520647 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
Call for Papers in Thematic Issues
Programmed Cell Death Genes in Oncology: Pioneering Therapeutic and Diagnostic Frontiers (BMS-CGT-2024-HT-45)
Programmed Cell Death (PCD) is recognized as a pivotal biological mechanism with far-reaching effects in the realm of cancer therapy. This complex process encompasses a variety of cell death modalities, including apoptosis, autophagic cell death, pyroptosis, and ferroptosis, each of which contributes to the intricate landscape of cancer development and ...read more
Related Journals
- Author Guidelines
- Graphical Abstracts
- Fabricating and Stating False Information
- Research Misconduct
- Post Publication Discussions and Corrections
- Publishing Ethics and Rectitude
- Increase Visibility of Your Article
- Archiving Policies
- Peer Review Workflow
- Order Your Article Before Print
- Promote Your Article
- Manuscript Transfer Facility
- Editorial Policies
- Allegations from Whistleblowers
- Announcements
Related Articles
-
Metabotropic Glutamate Receptors: A Review on Prospectives and Therapeutic Aspects
Mini-Reviews in Medicinal Chemistry Patent Selections
Recent Patents on Anti-Cancer Drug Discovery Cervical Cancer Diagnosis: Insights into Biochemical Biomarkers and Imaging Techniques
Combinatorial Chemistry & High Throughput Screening Oxidative Phosphorylation as a Target to Arrest Malignant Neoplasias
Current Medicinal Chemistry Role of Alterations in the Apoptotic Machinery in Sensitivity of Cancer Cells to Treatment
Current Pharmaceutical Design The Promise of Slow Down Ageing May Come from Curcumin
Current Pharmaceutical Design Recent Development in Nano-Sized Dosage Forms of Plant Alkaloid Camptothecin-Derived Drugs
Recent Patents on Anti-Cancer Drug Discovery Elastin-Like Recombinamers As Smart Drug Delivery Systems
Current Drug Targets Gas1 is a Pleiotropic Regulator of Cellular Functions: from Embryonic Development to Molecular Actions in Cancer Gene Therapy
Mini-Reviews in Medicinal Chemistry Morphology of C6 Glioma Cells on a Water-Repellent Fractal Alkyl Ketene Dimer Surface
Current Nanoscience Combination Treatment of Glioblastoma by Low-Dose Radiation and Genistein
Current Radiopharmaceuticals The Quinoline Imidoselenocarbamate EI201 Blocks the AKT/mTOR Pathway and Targets Cancer Stem Cells Leading to a Strong Antitumor Activity
Current Medicinal Chemistry Tubulins as Therapeutic Targets in Cancer: from Bench to Bedside
Current Pharmaceutical Design microRNA Biogenesis Pathway as a Therapeutic Target for Human Disease and Cancer
Current Pharmaceutical Design Applications of Polymeric Nanocapsules in Field of Drug Delivery Systems
Current Drug Discovery Technologies Yeast as a Powerful Model System for the Study of Apoptosis Regulation by Protein Kinase C Isoforms
Current Pharmaceutical Design Diagnosis and Management of Endocrine Hypertension in Children and Adolescents
Current Pharmaceutical Design Development of HIV Reservoir Targeted Long Acting Nanoformulated Antiretroviral Therapies
Current Medicinal Chemistry Modulation of intracellular pH in human ovarian cancer.
Current Molecular Medicine Nutraceuticals and "Repurposed" Drugs of Phytochemical Origin in Prevention and Interception of Chronic Degenerative Diseases and Cancer
Current Medicinal Chemistry